ASGCT Meeting Highlights Breakthrough Gene Therapies and Industry Developments

The American Society of Gene and Cell Therapy (ASGCT) annual meeting in New Orleans showcased cutting-edge research and clinical findings with far-reaching implications for medical practice. From personalized CRISPR therapies to advancements in treating rare diseases, the conference highlighted significant progress in the field of gene and cell therapy.

World's First Personalized CRISPR Therapy Shows Promise

In a groundbreaking development, researchers reported the first-ever case of a personalized in vivo CRISPR editing therapy, which substantially eased symptom burden in an infant with severe carbamoyl-phosphate synthetase 1 (CSP-1) deficiency. The case, published in the New England Journal of Medicine, involved a male infant who received two infusions of a customized CRISPR-based gene editing therapy at 7 and 8 months of age.

Following treatment, the patient showed remarkable improvements, including the ability to gradually increase dietary protein intake and reduced blood ammonia levels. Doctors were also able to scale back nitrogen-scavenging medication. While the therapy demonstrated a manageable safety profile, researchers noted transient elevations in liver enzymes and two viral infections within 4 weeks after the second dose.

Rocket Pharmaceuticals and Sarepta Therapeutics Present Promising Data

Rocket Pharmaceuticals revealed encouraging data for its investigational gene therapy RP-A601 in patients with plakophilin-2 related arrhythmogenic cardiomyopathy. The therapy normalized right ventricle systolic function and suppressed or stabilized off-beat contractions of the ventricles. Patients also reported improved quality of life and functional performance. Despite a serious adverse event in one patient, BMO Capital Markets analysts deemed the overall benefit/risk profile favorable, given the lack of approved disease-modifying treatments in this space.

Sarepta Therapeutics presented consolidated cardiovascular outcomes for its Duchenne muscular dystrophy gene therapy, Elevidys, from four studies totaling 218 patients. The data showed a manageable cardiac safety profile over five years of follow-up, with fluctuations in cardiac troponin levels being largely asymptomatic. Two cases of myocarditis were reported but resolved within three weeks. Additionally, Sarepta provided data on Elevidys in ambulatory children aged 8 to 9 years, demonstrating statistically significant and clinically meaningful functional improvements.

Neurogene Implements New Safety Protocol Following Patient Death

Neurogene unveiled a new safety monitoring and treatment system for its Rett Syndrome gene therapy, NGN-401, following a patient death in its Phase I/II study. The company's enhanced protocol includes daily surveillance in the first week post-treatment, focusing on fever, falling blood counts, and elevated ferritin levels. Neurogene has also implemented standard algorithms to treat systemic hyperinflammation, including high-dose corticosteroid intervention followed by an IL-1 receptor agonist.

The company is proceeding with the lower 1E15 vg dose of NGN-401 in Phase I/II development, reporting no known cases of systemic hyperinflammation at this dose level. This development underscores the ongoing challenges and evolving safety considerations in gene therapy research.